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GPT2 silencing

Development stage
Preclinical
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous, Subcutaneous, Intrathecal
01

Overview

GPT2 silencing refers to the targeted inhibition or downregulation of the gene encoding Glutamic--Pyruvic Transaminase 2 (GPT2), a mitochondrial enzyme also known as alanine aminotransferase 2. This enzyme catalyzes the reversible transamination between alanine and 2-oxoglutarate to generate pyruvate and glutamate, playing a key role in gluconeogenesis and amino acid metabolism in tissues such as liver, kidney, and skeletal muscle[2]. Silencing of GPT2 is typically achieved using RNA interference technologies (such as siRNA or shRNA) or antisense oligonucleotides. The primary mechanism involves reducing expression of the GPT2 gene to decrease gluconeogenesis from amino acids—an approach that has been investigated for metabolic diseases like diabetes[1][2]. There are no approved drugs specifically named "GPT2 silencing"; rather, this term describes an experimental therapeutic strategy.

Other names
GPT2 knockdownGPT-2 knockdownGPT 2 knockdownalanine transaminase 2 silencingglutamic-pyruvic transaminase 2 silencing
02

Targets

Glutamate pyruvate transaminase 2 mRNA

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